View Public Comments for CED Public Solicitation

Commenter:

Etheredge, Lynn

Title:

Rapid Learning Project

Organization:

George Washington University

Date:

12/26/2011

Comment:

Thank you for the invitation to comment on Medicare's CED policies. I believe it is essential to learn as much as possible, as soon as possible, about important new medical technologies and their best uses. A national CED process can be a vital element of such a policy initiative. I have laid out a proposal in a study "A High Performance System for Comparative Effectiveness Research" that appeared in Health Affairs (Oct. 2010, 29:10 (1761-1767) that i will forward to the e-mail address below. For example, the article says
"A Rapid-Learning System For New
Technologies"
The assessment of new medical technologies—
defined broadly to include drugs, devices, biologics,
diagnostics, and procedures—poses important
challenges for a new comparative
effectiveness research system. So far, the most
important advance in furthering national capabilities
for research into new technologies has
been the FDA Sentinel Initiative.
Building On The FDA Sentinel Initiative
Launched in 2008, this program will eventually
give the government access to up to 100 million
patient records for surveillance of drug safety
problems. It has been largely a reactive, safetyfocused
mechanism that awaits the first signals
of possible trouble and only then sets off a wider
review of databases from health plans and other
sources. But the risks of a wait-and-see approach
are that it will miss important clues that can and
should have been detected faster, and that the
data sources will prove to be incomplete or noncomparable
so that they cannot provide definitive
answers.
For rapid learning in comparative effectiveness
research, the Sentinel Initiative should be
expanded beyond its initial safety focus to systematically
collect data on therapies’ effectiveness,
starting when a new therapy first enters
clinical practice. Indeed, one of the most important
unanswered questions about new therapies
is this:What are its benefits compared to those of
existing therapies for real-world patients—including
(for an individual patient) patients like
me?
A national comparative effectiveness research
program needs an upgraded data collection system
that will build on Sentinel’s capabilities and
use a national set of metrics to compare the effectiveness
of alternative technologies. The new
system would aim to learn as much as possible—
and as soon as possible—about the optimal use of
new technologies. Even less is known about new
technologies than about old ones. As the
Congressional Budget Office (CBO) has pointed
out, the health sector’s adoption of new technologies
is a leading driver of health care spending
and the federal deficit.4
Timing The period after a new technology is
introduced is likely to offer a rich learning opportunity.
During this stage, clinicians use new
technologies on a much more diverse group of
patients—and for more conditions and combinations
of conditions—than are covered in the
clinical trials. This contrasts with the premarket
period, where the FDA allows studies on selected
groups and illnesses. A larger and broader patient
base will also create a richer data resource,
allowing investigators to identify and study
risks, benefits, and other issues for subgroups
with greater confidence.
In a landmark study that identified important
preapproval knowledge gaps that research
should fill in, Annetine Gelijns, Nathan
Rosenberg, and Alan Moskowitz note: “The
history of medical innovation is replete with instances
in which new indications have been discovered
after drugs and devices have been
introduced into clinical practice.” Their study
reports, for example, that as of 1993, 90 percent
of “blockbuster” drugs had important indications
beyond those for which they were originally
approved. By 1995, secondary uses
accounted for more than 40 percent of their
sales. The study found a similar pattern among
medical devices.5(p694)
Similarly, the National Comprehensive Cancer
Network estimated in 2005 that 50–75 percent of
all prescriptions for cancer therapies were “offlabel,”
defined as the use of a drug for purposes
other than those for which the federal
government found proof of effectiveness and
safety.6 By some estimates, more than half of
prescription drug use is now off-label. For surgical
procedures, the study by Gelijns and colleagues
reported that only 4–13 percent of
patients undergoing coronary bypass surgery
would have met the eligibility criteria for the
clinical trial that established its efficacy.5 A com-
parative effectiveness research initiative for new
technologies could fill in these kinds of knowledge
gaps quickly.
How The System Would Work A national
learning system fornew technologies could work
as follows. At the point of market approval or the
start of Medicare payments—in the case of surgical
procedures—HHS, in consultation with the
private sector, would establish a national research
plan for the technology or class of technology
in question. The plan would specify
metrics for comparing effectiveness; propose
key questions about appropriate uses, effectiveness,
and safety; create national reporting registries;
and assign funding responsibility for
studies.
At the end of an initial period—for example,
three years—expert panels would review the evidence
and determine future research needs.
Medicare, Medicaid, and private-sector payers
would adopt common “coverage with evidence
development” policies to require reporting of
needed data from health care providers to answer
key research questions as a condition for
payment. To design and implement this comparative
effectiveness research study system, the
federal government should work with physicians,
patients, researchers, the new Patient-
Centered Outcomes Research Institute, health
insurance plans, and pharmaceutical and biotechnology
companies.
In starting this new-technologies comparative
effectiveness research initiative, the federal
government should set research priorities such
as new cancer treatments, new surgical procedures,
and off-label uses of drugs.With hundreds
of cancer drugs under development, it will be
important to learn about these treatments as
quickly and definitively as possible. Capturing
off-label uses of new drugs by cancer patients
will also be an important advance.
The federal government does not require
safety and efficacy studies for new surgical procedures,
so there is often much uncertainty, even
within the surgical professions, about their comparative
benefits and risks. Surgical procedures
also account for far more medical spending than
do drugs. And patients face difficult choices between
standard surgery, laparoscopic surgery,
drug treatment, or lifestyle changes, and they
deserve to be well informed.
As noted in the article by Gelijns and colleagues,
it is particularly important to learn
quickly about secondary uses for new drugs, in
part because they have been important sources
for advancing the therapeutic arsenal, and also
because of potential problems that may arise,
such as higher death rates (as happened with
Vioxx, a nonsteroidal anti-inflammatory drug
that had to be withdrawn from the market after
it had been approved). Physicians, patient
groups, and the pharmaceutical industry should
collaborate in these studies.